Around the Helix: Cell and Gene Therapy Company Updates – March 9, 2022

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

1. LEXEO Gene Therapy Decreases Alzheimer Disease Biomarkers

LEXEO Therapeutics’ LX1001 decreased core Alzheimer disease (AD) biomarkers in patients treated with the gene therapy, according to data from the first cohort of its phase 1/2 clinical trial (NCT03634007).

2. New Startup Hopes to Streamline Gene Therapy Development

Andson Biotech is a new startup launched from a partnership between the Georgia Institute of Technology and the National Science Foundation Engineering Research Center for Cell Manufacturing Technologies. The company’s mass spectrometry approach can identify and measure biomolecules in minutes.

3. Myasthenia Gravis CAR T-Cell Therapy Receives Fast Track Designation

Cabaletta Bio's muscle-specific kinase (MuSK) chimeric autoantibody receptor T (MuSK-CAART) therapy has received fast track designation for improving activities of daily living and muscle strength in patients with myasthenia gravis.

4. Novartis Licenses Voyager AAV Vectors

Novartis and Voyager Therapeutics have entered into a licensing agreement to allow Novartis access to Voyager’s next-generation AAV capsids for potential therapy development in central nervous system indications. The agreement may be expanded to include 2 additional targets.

5. New Collaboration to Discover Novel Antibodies for Gene Therapy Delivery

Kriya Therapeutics and Twist Bioscience are collaborating to discover novel antibodies to deliver with AAV gene therapy in oncologic indications. The agreement leverages Twist’s antibody discovery and optimization platform and Kriya’s proprietary gene therapy technology platform.

6. Novel Gene Therapy for Overactive Bladder Yields Clinically Meaningful Symptom Reduction

Urovant Sciences announced topline results from their phase 2a trial (NCT04211831) of URO-902, their novel plasmid human cDNA encoding maxi-K channel gene therapy. The therapy was well-tolerated and treated participants experienced clinically meaningful and statistically significant reductions in micturitions and urgency episodes as well as improvements in quality-of-life indicators compared with placebo 12 weeks post-treatment.

7. WuXi Advanced Therapies Launches New Gene Therapy Manufacturing Technology

The company, a subsidiary of WuXi AppTec, launched the Tetracycline-Enabled Self-Silencing Adenovirus (TESSA) platform, which is a stateoftheart novel process designed for the scalable, transfection-free, Good Manufacturing Practice grade manufacture of AAV vectors. The platform should expedite, streamline, and reduce costs of AAV manufacturing.

8. Mesenchymal Stem Cell Improves Joint Function in Rheumatoid Arthritis

Hope Biosciences’ adipose-derived mesenchymal stem cell therapy, HB-adMSCs, has demonstrated safety and efficacy in rheumatoid arthritis in a phase 1/2 pilot study (NCT03691909). The therapy yielded significant and durable joint function improvements as well as improvements in inflammatory marker C-reactive protein.

9. WVU Cancer Institute Cellular Therapy Program Receives FACT Accreditation

The institute’s blood and marrow transplant program was reaccredited for the sixth consecutive time and their immune effector, CAR T-cell therapy received its first Foundation for the Accreditation of Cellular Therapy (FACT) accreditation, demonstrating that these programs exceed standards for patient care and laboratory services.

10. Future Uncertain for bluebird bio

The gene therapy company held a conference call in which they shared that their chief financial officer had resigned as well as their "substantial doubt" about how long operations will continue due to negative cash flow.